C9orf72

May 11, 2018May 11, 2018

MIT Researchers Find Potential Role of C9orf72 Gene in ALS, Frontotemporal Dementia

News

Mutations in the C9orf72 gene deregulate the process by which cells remove waste, leading to a buildup of toxic elements inside them, researchers have found. This discovery expands knowledge ... Read more

February 8, 2018

Restoring Missing Protein Stops Nerve Cell Degeneration, ALS Study Shows

News

Restoring a protein that’s missing in a type of ALS that affects 10 percent of patients stopped nerve cell deterioration, University of Southern California researchers report. The results also ... Read more

January 16, 2018

Easing the Nerve Cell Traffic Jam May Be a Way to Treat ALS, Research Suggests

News

A traffic jam inside nerve cells is a feature of most forms of amyotrophic lateral sclerosis and frontotemporal dementia, a study shows. It suggests that easing the jam is ... Read more

January 5, 2018

Sangamo and Pfizer to Collaborate on Developing Gene Therapies for ALS

News

Sangamo Therapeutics and Pfizer will work together on gene therapies they hope will overcome mutations that cause ALS and frontotemporal lobar degeneration. Gene therapy works by delivering a healthy copy ... Read more

January 4, 2018January 4, 2018

Cell Stress Unleashes Downward Spiral of Toxic Protein Production in ALS

News

Cell stress, such as a viral infection, may trigger the production of faulty proteins from the C9orf72 gene, a leading contributor of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia ... Read more

December 22, 2017

Gene Editing Shows Promise in Treating Familial ALS, Mouse Study Suggests

News

Gene editing may be a viable tool to slow disease-causing processes in amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, a mouse study indicates. Published in ... Read more

December 19, 2017December 19, 2017

Wave Life Sciences Therapy Reduces Levels of ALS Markers, Animal Studies Show

News

The Wave Life Sciences therapy WVE-3972-01 reduced markers of the form of ALS that scientists believe stems from a gene mutation, studies show. Researchers said the potential treatment blocked faulty versions ... Read more

November 30, 2017November 30, 2017

Rare Mutations Can Interact With Related Proteins to Promote ALS Progression, Study Shows

News

Rare genetic mutations that affect functionally related proteins act in a synergistic manner to determine the course of ALS, a new genetic screening study shows. It also points to ... Read more

October 26, 2017

Relatives of ALS Patients More Likely to Develop Neuropsychiatric Disorders, Study Finds

News

Relatives of patients with amyotrophic lateral sclerosis (ALS) have a higher likelihood of developing neuropsychiatric symptoms including schizophrenia, obsessive-compulsive disorder, autism, and alcoholism, research from Ireland shows. The study, ... Read more

August 28, 2017August 28, 2017

Wave Is Developing Two Drugs That Target a Common ALS Mutation

News

Wave Life Sciences is developing two ALS and frontotemporal dementia therapies that target a gene mutation common in both. The therapies target mutations of the C9orf72 gene, Wave said in ... Read more

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